Oxymetholone

Medically reviewed by Drugs.com. Last updated on Jun 19, 2023.

Pronunciation

(oks i METH oh lone)

Dosage Forms

Excipient information presented when available (limited, particularly for generics); consult specific product labeling.

Tablet, Oral:

Anadrol-50: 50 mg [scored]

Brand Names: U.S.

Anadrol-50

Pharmacologic Category

Anabolic Steroid

Pharmacology

Enhances production of erythropoietin in patients with anemias which are due to bone marrow failure; stimulates erythropoiesis in anemias due to deficient red cell production

Onset of Action

Response is not often immediate; a minimum trial of 3 to 6 months is recommended

Use: Labeled Indications

Anemia: Treatment of anemias caused by deficient red cell production. Note: Androgen therapy (eg, oxymetholone) is generally not appropriate for the treatment of anemias except in certain rare situations (eg, Fanconi anemia).

Off Label Uses

Fanconi anemia

Data from a limited number of patients in a retrospective review suggests that oxymetholone may be beneficial in the treatment of Fanconi anemia ^{[Paustian 2016]}. However, due to significant risks associated with therapy, a careful risk vs. benefit assessment is advised. Additional data may be necessary to further define the role of oxymetholone in this condition.

Contraindications

Hypersensitivity to oxymetholone or any component of the formulation; breast cancer in men; breast cancer in women with hypercalcemia; prostate cancer; severe hepatic dysfunction; nephrosis or nephrotic phase of nephritis; pregnancy or use in women who may become pregnant

Dosing: Adult

Erythropoietic effects: Oral: 1 to 5 mg/kg/day once daily; usual effective dose: 1 to 2 mg/kg/day; give for a minimum trial of 3 to 6 months because response may be delayed

Fanconi anemia (off-label use): Limited data available: Oral: Initial: 2 mg/kg/day (median dose reported); most patients had a documented dose reduction although exact doses were not provided

Duration of therapy: 4.1 years (median for responders); 1.3 years (median for non-responders) (Paustian 2016). Additional data may be necessary to further define the role of oxymetholone in this condition.

Dosing: Geriatric

Refer to adult dosing.

Dosing: Pediatric

Anemia, treatment (erythropoietic effect [eg, Fanconi anemia, bone marrow failure syndromes]):

Children and Adolescents: Oral: Initial: ~2 mg/kg/day, rounded to nearest 12.5 mg (ie, ¹/₄ of the 50 mg tablet); usual range: 1 to 5 mg/kg/day once daily; some have suggested higher dosing initially (2 to 5 mg/kg/day) and after response, a slow taper (eg, decrease dose by 10% to 20% every 3 to 4 months) to lowest effective dose with minimal side effects; typically, therapeutic response of stabilization or improvement in hemoglobin or platelet counts is observed within 3 to 6 months (Camitta 1979; FARF 2014). Note: The National Kidney Foundation recommends against the use of androgens as an adjuvant to erythropoiesis-stimulating agent treatment in anemic patients with chronic kidney disease (KDIGO 2012; KDOQI 2006). Although an FDA-approved indication, oxymetholone use in the treatment of aplastic anemia has been replaced by newer therapies with improved outcomes (eg, hematopoietic stem cell transplantation, immunosuppressive therapy) (BSH [Samarasinghe 2017]; NAPAAC [Rogers 2019]).

Storage

Store at room temperature 20°C to 25°C (68°F to 77°F); excursions permitted to 15°C to 30°C (59°F to 86°F). Protect from light.

Drug Interactions

Agents with Blood Glucose Lowering Effects: Androgens may enhance the hypoglycemic effect of Agents with Blood Glucose Lowering Effects. Monitor therapy

Ajmaline: Androgens may enhance the adverse/toxic effect of Ajmaline. Specifically, the risk for cholestasis may be increased. Monitor therapy

C1 inhibitors: Androgens may enhance the thrombogenic effect of C1 inhibitors. Monitor therapy

Corticosteroids (Systemic): May enhance the fluid-retaining effect of Androgens. Monitor therapy

CycloSPORINE (Systemic): Androgens may enhance the hepatotoxic effect of CycloSPORINE (Systemic). Androgens may increase the serum concentration of CycloSPORINE (Systemic). Management: Consider avoiding concomitant use of androgens and cyclosporine. If concomitant use is unavoidable, monitor serum cyclosporine concentrations and for signs and symptoms of hepatotoxicity. Cyclosporine dose reductions may be required. Consider therapy modification

Vitamin K Antagonists (eg, warfarin): Androgens may enhance the anticoagulant effect of Vitamin K Antagonists. Management: Monitor for increased effects of vitamin K antagonists if an androgen is initiated/dose increased, or decreased effects if androgen is discontinued/dose decreased. Significant reductions in vitamin K antagonist dose are likely required. Consider therapy modification

Test Interactions

Decreased thyroxine-binding globulin, T₄; increased resin uptake of T₃ and T₄

Adverse Reactions

The following adverse drug reactions and incidences are derived from product labeling unless otherwise specified.

Frequency not defined:

Cardiovascular: Coronary artery disease, peripheral edema

Central nervous system: Chills, deepening of the voice (females), excitement, insomnia

Dermatologic: Acne vulgaris, androgenetic alopecia (postpubertal males, females), hyperpigmentation

Endocrine & metabolic: Amenorrhea, change in libido (decreased/increased), decreased glucose tolerance, decreased HDL cholesterol, gynecomastia, hirsutism (women), hypercalcemia, hyperchloremia, hyperkalemia, hypernatremia, hyperphosphatemia, increased LDL cholesterol, menstrual disease

Gastrointestinal: Diarrhea, nausea, vomiting

Genitourinary: Benign prostatic hypertrophy (elderly males), clitoromegaly, decreased ejaculate volume, epididymitis, erectile dysfunction (increased erections; prepubertal males), impotence, irritable bladder, oligospermia, phallic enlargement, priapism, testicular atrophy, testicular disease, virilization (females)

Hematologic & oncologic: Clotting factors suppression (II, V, VII, X), hemorrhage, increased INR, iron deficiency anemia, leukemia, malignant neoplasm of prostate, prolonged prothrombin time

Hepatic: Cholestatic hepatitis, cholestatic jaundice, hepatic failure, hepatic necrosis, hepatocellular neoplasm (including carcinoma), increased serum alkaline phosphatase, increased serum bilirubin, increased serum transaminases, peliosis hepatitis

Neuromuscular & skeletal: Increased creatine phosphokinase, premature epiphyseal closure (children)

Renal: Increased serum creatinine

Respiratory: Hoarseness (females)

<1%, postmarketing, and/or case reports: Hepatotoxicity (idiosyncratic; Chalasani 2014)

ALERT: U.S. Boxed Warning

Peliosis hepatis:

Peliosis hepatis, a condition in which liver and, sometimes, splenic tissue is replaced with blood-filled cysts, has occurred in patients receiving androgenic anabolic steroids. These cysts are sometimes present with minimal hepatic dysfunction and have been associated with liver failure. Often, they are not recognized until life-threatening liver failure or intra-abdominal hemorrhage develops. Withdrawal of drug usually results in complete disappearance of lesions.

Liver cell tumors:

Most often these tumors are benign and androgen-dependent, but fatal malignant tumors have occurred. Withdrawal of drug often results in regression or cessation of tumor progression. However, hepatic tumors associated with androgens or anabolic steroids are much more vascular than other hepatic tumors and may be silent until life-threatening, intra-abdominal hemorrhage develops.

Blood lipid changes:

Blood lipid changes associated with increased risk of atherosclerosis are seen in patients treated with androgens and anabolic steroids. These changes include decreased high-density lipoprotein (HDL) and, sometimes, increased low-density lipoprotein (LDL). The changes may be very marked and could have a serious impact on the risk of atherosclerosis and coronary artery disease.

Warnings/Precautions

Concerns related to adverse effects:

• Blood lipid changes: [US Boxed Warning]: Anabolic steroids may cause changes in blood lipids (decreased high density lipoproteins and sometimes increased low density lipoproteins), increasing the risk of arteriosclerosis and coronary artery disease.

• Clotting factor alterations: Anabolic steroids may suppress factors II, V, VII, and X; prothrombin time may be increased.

• Hepatic effects: [US Boxed Warning]: Androgenic anabolic steroid treatment may cause peliosis hepatis, which occurs when splenic or hepatic tissue is replaced by cysts (blood-filled); may only cause minimal hepatic dysfunction although has been associated with hepatic failure. Androgenic liver cell tumors, which may be benign, although malignant tumors have also been reported; generally regress when anabolic steroid treatment is withdrawn. Both conditions (peliosis and tumors) may not be apparent until liver failure or intra-abdominal hemorrhage develops. Androgen use (low doses) has also been associated with cholestatic hepatitis and jaundice; may be associated with hepatomegaly and right upper-quadrant pain; jaundice is typically reversible upon discontinuation (continuing treatment has been associated with coma and death). Monitor liver function periodically.

• Prostate conditions: Androgenic anabolic steroid use may cause prostatic hypertrophy or prostate cancer in elderly men.

Disease-related concerns:

• Breast cancer: May cause hypercalcemia in women with breast cancer by stimulating osteolysis.

• Diabetes: Use with caution in patients with diabetes mellitus; insulin or oral hypoglycemic needs may be altered; monitor carefully.

• Edematous conditions: Use with caution in patients with conditions influenced by edema (eg, cardiovascular disease, migraine, seizure disorder, renal impairment); may cause fluid retention.

Special populations:

• Pediatric: May accelerate bone maturation without producing compensatory gain in linear growth in children; effect may continue for 6 months after treatment discontinuation; in prepubertal children perform radiographic examination of the hand and wrist every 6 months to determine the rate of bone maturation and to assess the effect of treatment on the epiphyseal centers.

• Women: Discontinue with evidence of mild virilization in women.

Other warnings/precautions:

• Appropriate use: Oxymetholone should not replace other anemia treatment supportive measures such as transfusion, correction of iron, folic acid, vitamin B₁₂ or pyridoxine deficiency, antibacterial therapy, and the appropriate use of corticosteroids.

Monitoring Parameters

Periodic liver function tests, lipid profile, hemoglobin and hematocrit; iron studies; serum glucose (may be decreased by testosterone, monitor patients with diabetes); radiologic examination of bones every 6 months (when using in prepubertal children); monitor urine and serum calcium and signs of virilization in women treated for breast cancer

Reproductive Considerations

Use is contraindicated in women who may become pregnant.

Oligospermia or amenorrhea may occur resulting in an impairment of fertility.

Pregnancy Risk Factor

Pregnancy Considerations

Use is contraindicated in women who are pregnant.

Patient Education

What is this drug used for?

• It is used to treat anemia.

All drugs may cause side effects. However, many people have no side effects or only have minor side effects. Call your doctor or get medical help if any of these side effects or any other side effects bother you or do not go away:

• Diarrhea

• Trouble sleeping

• Sexual dysfunction

WARNING/CAUTION: Even though it may be rare, some people may have very bad and sometimes deadly side effects when taking a drug. Tell your doctor or get medical help right away if you have any of the following signs or symptoms that may be related to a very bad side effect:

• Liver problems like dark urine, feeling tired, lack of appetite, nausea, abdominal pain, light-colored stools, vomiting, or yellow skin or eyes.

• Erection that lasts more than 4 hours

• Acne

• Shortness of breath

• Excessive weight gain

• Swelling of arms or leg

• Unable to pass urine

• Change in amount of urine passed

• Muscle weakness

• Bruising

• Bleeding

• Severe anxiety

• Virilization like in females a deep voice, facial hair, pimples, or period changes.

• Signs of an allergic reaction, like rash; hives; itching; red, swollen, blistered, or peeling skin with or without fever; wheezing; tightness in the chest or throat; trouble breathing, swallowing, or talking; unusual hoarseness; or swelling of the mouth, face, lips, tongue, or throat.

Note: This is not a comprehensive list of all side effects. Talk to your doctor if you have questions.

Consumer Information Use and Disclaimer: This information should not be used to decide whether or not to take this medicine or any other medicine. Only the healthcare provider has the knowledge and training to decide which medicines are right for a specific patient. This information does not endorse any medicine as safe, effective, or approved for treating any patient or health condition. This is only a limited summary of general information about the medicine's uses from the patient education leaflet and is not intended to be comprehensive. This limited summary does NOT include all information available about the possible uses, directions, warnings, precautions, interactions, adverse effects, or risks that may apply to this medicine. This information is not intended to provide medical advice, diagnosis or treatment and does not replace information you receive from the healthcare provider. For a more detailed summary of information about the risks and benefits of using this medicine, please speak with your healthcare provider and review the entire patient education leaflet.